PL3411484T3 - Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego - Google Patents

Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego

Info

Publication number
PL3411484T3
PL3411484T3 PL17747935.9T PL17747935T PL3411484T3 PL 3411484 T3 PL3411484 T3 PL 3411484T3 PL 17747935 T PL17747935 T PL 17747935T PL 3411484 T3 PL3411484 T3 PL 3411484T3
Authority
PL
Poland
Prior art keywords
adenosatellite
virus
complementary
threaded
injection
Prior art date
Application number
PL17747935.9T
Other languages
English (en)
Inventor
Anthony DONSANTE
Karen Kozarsky
Nicholas Matthew Boulis
Jonathan Patrick Riley
Original Assignee
Emory University
Regenxbio Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Emory University, Regenxbio Inc. filed Critical Emory University
Publication of PL3411484T3 publication Critical patent/PL3411484T3/pl

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL17747935.9T 2016-02-05 2017-01-25 Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego PL3411484T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662292157P 2016-02-05 2016-02-05
PCT/US2017/014914 WO2017136202A1 (en) 2016-02-05 2017-01-25 Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

Publications (1)

Publication Number Publication Date
PL3411484T3 true PL3411484T3 (pl) 2024-02-19

Family

ID=59500060

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17747935.9T PL3411484T3 (pl) 2016-02-05 2017-01-25 Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego

Country Status (15)

Country Link
US (3) US12070510B2 (pl)
EP (2) EP4299079A3 (pl)
JP (4) JP2019504888A (pl)
AU (1) AU2017214283B2 (pl)
DK (1) DK3411484T3 (pl)
ES (1) ES2962860T3 (pl)
FI (1) FI3411484T3 (pl)
HR (1) HRP20231451T1 (pl)
HU (1) HUE064408T2 (pl)
IL (2) IL300254B2 (pl)
LT (1) LT3411484T (pl)
PL (1) PL3411484T3 (pl)
PT (1) PT3411484T (pl)
SI (1) SI3411484T1 (pl)
WO (1) WO2017136202A1 (pl)

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US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
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CN112553210B (zh) 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
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BR112021006060A2 (pt) 2018-10-01 2021-07-20 The Trustees Of The University Of Pennsylvania composições úteis para tratamento de gangliosidose gm1
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JP2022525564A (ja) * 2019-03-21 2022-05-17 ピーティーシー セラピューティクス， インコーポレイテッド アンジェルマン症候群を治療するためのベクターおよび方法
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Also Published As

Publication number Publication date
JP2024084797A (ja) 2024-06-25
EP4299079A3 (en) 2024-05-01
HRP20231451T1 (hr) 2024-03-01
IL300254B1 (en) 2025-02-01
IL260954A (pl) 2018-09-20
EP4299079A2 (en) 2024-01-03
WO2017136202A1 (en) 2017-08-10
IL260954B2 (en) 2023-07-01
ES2962860T3 (es) 2024-03-21
LT3411484T (lt) 2023-11-27
IL300254B2 (en) 2025-06-01
SI3411484T1 (sl) 2023-12-29
EP3411484B1 (en) 2023-10-04
IL300254A (en) 2023-03-01
EP3411484A1 (en) 2018-12-12
US12070510B2 (en) 2024-08-27
US20190038777A1 (en) 2019-02-07
JP2025041933A (ja) 2025-03-26
JP2019504888A (ja) 2019-02-21
FI3411484T3 (fi) 2023-11-15
CA3012653A1 (en) 2017-08-10
IL260954B1 (en) 2023-03-01
HUE064408T2 (hu) 2024-03-28
US20250135041A1 (en) 2025-05-01
AU2017214283B2 (en) 2023-06-29
AU2017214283A1 (en) 2018-08-02
EP3411484A4 (en) 2019-08-07
US20240108761A1 (en) 2024-04-04
JP2022101648A (ja) 2022-07-06
PT3411484T (pt) 2023-11-17
DK3411484T3 (da) 2023-11-20

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